Results

Forty four patients with ileocolonic involvement underwent stem cell mobilisation before randomisation to Early (n=23, all treated) or Delayed (n=21) HSCT. One Early HSCT patient died 14 days after HSCT. One randomised to Delayed HSCT withdrew immediately, 4 did not proceed because they required surgery (n=3) or were too well after 1 year (n=1). Sixteen patients underwent delayed HSCT. The CDAI fell from 321 (median, IQR 242-433) to 162 (76-294) one year after active treatment compared to 353 (300-462) to 310 (236-403) over year 1 prior to Delayed HSCT. At year 2 the CDAI was maintained at 161 (59-232) in Early HSCT patients and had fallen to 165 (88-298) in Delayed HSCT patients. The SES-CD was 13 (8.5-24.5) at baseline, 3 (1-10) after one year and 3 (0-9) at two years in early HSCT t patients compared to 13 (6.5-18), 7 (4-20) and 2 (0-7) in delayed HSCT patients. Ten patients (baseline SES-CD score of 7 [5-13.5]) became free of endoscopic evidence of active or inactive Crohn's disease (SES-CD = 0) one year after HSCT compared to none following control treatment. Three of 4 remained disease free and 4 more became free of endoscopic disease in year two after HSCT. The CDAI was <150 in 16 patients one year after HSCT vs one of 21 following control treatment. IBDQ scores at baseline, 1 and 2 years were 129 (103-142), 153 (121-203) and 173 (126-211) in the early HSCT group compared to 109 (83-137), 124 (87-154) and 148 (113-195) in the delayed HSCT group. Comparable values for the EuroQuol VAS score were 61 (40-66), 80 (62-88) and 85 (63-90) in the early HSCT group vs 43 (31-63), 50 (35-70) and 80 (70-85) in the late HSCT group.

Conclusion

Improvements gained one year after HSCT are maintained into a second year. HSCT delayed by one year appears to be effective. There is a progressive improvement in quality of life. If improvements remain durable, the risks of HSCT might become acceptable for a wider group of patients.